Pilot trial of CTLA4-Ig (Abatacept) in a child affected by a severe congenital autoimmune syndrome (IPEX) - Trial of Abatacept in IPEX syndrome

Conditions: For patients with a stabilized clinical condition, not recovered in care units for acute problems.
MedDRA version: 9.1Level: HLGTClassification code 10003816
MedDRA version: 9.1Level: HLTClassification code 10027657

Registration Number: EUCTR2008-003368-21-IT

Lead Sponsor: ISTITUTO PER L`INFANZIA BURLO GAROFOLO

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: Male

Target Recruitment: Not specified

Inclusion Criteria

Confirmed diagnosis of IPEX. Unfavourable cost/benefit ratio for Hematopoietic Stem Cell Transplantation. In particular: 1)patients who already developed autoimmune disorders and/or organ failure because of the disease 2)disease not fully controlled by an intensive treatment at doses of conventional immunosuppressants producing severe side effects (infections, adrenal-hypotalamic suppression).
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Positivity for mycobacterius tuberculosis infection.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To improve the ratio between benefits and costs for the treatment with drugs in subjects not eligible for hematopoietic stem cell transplantation. In particular, to obtain a better control of the disease with less of the immunosuppressants-related side effects (infections and suppression of the hypothalamic axis).;Secondary Objective: To widen the trial and to propose new protocols for treating IPEX.;Primary end point(s): Survival without the occurrence of new autoimmune phenomena; number and severity of autoimmune flares (cutanous, intestinal, haematological, respiratory). Residual growth.

Secondary Outcome Measures